U.S. Government Accountability Office
Orphan drug sales have nearly doubled since 2008, a trend set to continue through 2022 as companies increasingly focus on rare disease meds.
The U.S. could run into a vaccine supply predicament if an influenza pandemic hits, since only one drugmaker has a domestic facility with egg-based vaccine production capabilities.
When PTC agreed to buy Marathon's Emflaza last week, it inherited congressional scrutiny on the drug's price, originally set at a much-lambasted $89,000.
U.S. government watchdogs plan to probe the FDA's orphan drug policies after Marathon's high-profile pricing scandal.
Generic drug prices in Medicare Part D decreased significantly in recent years. So why the worry about price hikes? "Extraordinary" increases on hundreds of meds, that's why.